December 12, 2022

The Duchenne Registry Celebrates 15 Years of Data Collection

The PPMD team is incredibly grateful for the families who have shared their Duchenne and Becker experience with The Duchenne Registry over the years. It is hard to believe that the first families joined the…

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December 8, 2022

Your Gift Today Funds ACTION

As a cardiologist, I think about the heart day and night. It is central and fundamental to all of us. We must protect and preserve the heart so that individuals with Duchenne or Becker can…

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December 6, 2022

Complete Your Registry Surveys and You Could Win Your Choice of a $150 Holiday Gift Card!

It’s the Holiday Season and we are thrilled to have another great raffle for families who participate in The Duchenne Registry! This December, the Registry will be giving away TWO $150 e-gift cards to the…

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December 5, 2022

Solid Biosciences Announces Completion of AavantiBio Acquisition & Update on Duchenne Pipeline Program

Today Solid Biosciences shared an update with the Duchenne community regarding their strategic development. Solid announced they have completed the acquisition of AavantiBio, a gene therapy company. The combined company will focus on advancing a…

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November 30, 2022

Race Athlete Spotlight: Jason Hendry

Jason Hendry is gearing up for his fourth race at the 2023 Walt Disney World Marathon® Weekend presented by State Farm. Meet Jason Why did you choose to join the Race to End Duchenne team?…

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November 28, 2022

Sarepta Announces Biologics License Application (BLA) Accepted For Filing by the FDA for SRP-9001

PPMD is thrilled to learn that the FDA has accepted for filing and granted priority review for the Biologics License Application (BLA) for Sarepta’s gene therapy product, SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant…

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November 23, 2022

This Thanksgiving I am Grateful for You.

As I prepare for Thanksgiving, I run through a quick inventory in my head. I’ve got turkey and stuffing, a pumpkin pie with lots of whipped cream, and of course, Diet Coke. Most importantly, family…

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November 21, 2022

PTC Shares Community Letter Updating on Activities To Bring Ataluren (Translarna) To US Patients

PTC Therapeutics has shared a community letter updating on the company’s activities to bring ataluren (Translarna) to US patients with nonsense mutations. PPMD will host a community webinar with PTC on Friday, December 9th at…

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November 18, 2022

Pfizer Announces First Participant Dosed in DAYLIGHT Phase 2 Gene Therapy Trial

Pfizer has announced that the first participant has been dosed in the Phase 2 DAYLIGHT study, which will evaluate the safety and tolerability of the company’s investigational mini-dystrophin gene therapy product (fordadistrogene movaparvovec) in very young…

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November 18, 2022

PPMD Provides Testimony During FDA CBER Meeting on Patient Perspectives on Gene Therapy Products

Earlier this week, Chief Advocacy Officer Ryan Fischer addressed the FDA’s Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) at a Public Patient-Focused Drug Development (PFDD) Meeting on Patient…

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